FDA Approves Drug For Rare Niemann-Pick Disease Type C
The genetic disorder, which is fatal, affects the nervous system and other organs — IntraBio's drug Aqneursa was shown to help reduce symptoms compared to a placebo. Separately, Amgen reported success for eczema and myasthenia gravis treatments.
Reuters:
US FDA Approves IntraBio's Drug For Rare Genetic Disease
The U.S. Food and Drug Administration approved IntraBio's drug for a rare and fatal genetic disorder, the health regulator said on Tuesday, just days after the agency cleared the first treatment for the disease. Niemann-Pick disease type C (NPC) is a rare genetic disorder that affects the nervous system and other organs, causing physical and mental disabilities such as speech issues, difficulties with swallowing and coordination, clumsiness and others over time. (9/24)
Stat:
Amgen Reports Success For Eczema And Myasthenia Gravis Treatments
Amgen on Tuesday reported that a pair of antibody drugs it is developing as treatments for immune-mediated diseases succeeded in late-stage trials, but the therapies did not clearly beat existing competitors on the market. (Wosen and DeAngelis, 9/24)
Axios:
AI's Latest Trick: Repurposing Old Drugs For Rare Disease
A new artificial intelligence tool could supercharge efforts to find new uses for old drugs, particularly rare diseases without a Food and Drug Administration-approved treatment. The Harvard Medical School researchers behind the tool, called TxGNN, write today in Nature Medicine that it can identify candidates for 17,000 conditions — the largest number of diseases that any single AI model can handle to date. (Reed, 9/25)
The New York Times:
Anti-Aging Enthusiasts Are Taking a Pill to Extend Their Lives. Will It Work?
In March, Robert Berger, 69, a self-proclaimed “better-living-through-chemistry type of person,” started taking a small dose of rapamycin once a week with the goal of increasing his “health span” — the amount of time he might live without serious disease. Rapamycin is typically prescribed to organ transplant patients to suppress their immune systems. But many scientists and longevity seekers like Mr. Berger think the drug can do much more than that: They say it can delay aging and age-related diseases. (Smith, 9/24)
